A recently completed clinical trial examining the use of genetic testing to direct cancer treatment was able to exceed its enrollment goal of 600 participants in less than 2 years instead of the expected 5 years. Patients were willing to participate even though they had to undergo an additional biopsy, revealing considerable interest in personalized treatment based on genetic tests. The trial confirmed that erlotinib (Tarceva) is highly effective in non-small cell lung cancer (NSCLC) patients with a mutation in the EGFR gene. It also found that NSCLC patients with mutations in the KRAS gene did not benefit from the novel cancer drug selumetinib. In contrast, not enough small cell lung cancer (SCLC) patients had any of the investigated mutations to properly test how they responded to treatments. Such mutations will require trials involving thousands of patients to draw reliable conclusions.

A recently completed clinical trial examining the use of genetic testing to direct cancer treatment was able to exceed its enrollment goal of 600 participants in less than 2 years instead of the expected 5 years. Patients were willing to participate even though they had to undergo an additional biopsy, revealing considerable interest in personalized treatment based on genetic tests. The trial confirmed that erlotinib (Tarceva) is highly effective in non-small cell lung cancer (NSCLC) patients with a mutation in the EGFR gene. It also found that NSCLC patients with mutations in the KRAS gene did not benefit from the novel cancer drug selumetinib. In contrast, not enough small cell lung cancer (SCLC) patients had any of the investigated mutations to properly test how they responded to treatments. Such mutations will require trials involving thousands of patients to draw reliable conclusions.

A clinical trial found that dabrafenib, a BRAF inhibitor, was far more effective in treating melanomas that have BRAF mutations than the chemotherapy drug dacarbazine, according to a report at an American Society of Clinical Oncology meeting. Patients treated with this drug lived without getting worse for 70% longer than those treated with dacarbazine (5.1 vs. 2.7 months, respectively). Moreover, compared to those treated with vemurafenib in other studies, dabrafenib-treated patients had less risk of another kind of skin cancer called squamous cell carcinoma. This suggests that dabrafenib, which is experimental, could be safer than vemurafenib, which is FDA approved.

A study in The Lancet shows that the drug ipilimumab could treat melanomas that have spread to the brain, particularly in people who do yet not have neurological symptoms. Of 51 such patients treated with ipilimumab, 12 had tumors in the brain that shrank or did not get worse and 14 had tumors outside the brain that shrank or did not get worse. Ipilimumab (Yervoy) is an immune system booster that the FDA has approved for treating advanced melanomas.

An experimental drug could help control some melanomas that have BRAF or NRAS mutations, according to a report at an American Society of Clinical Oncology meeting. Tumors shrank or did not get worse in 8 out of 35 patients with the most common BRAF mutation (V600E), and in 6 out of 28 patients with NRAS mutations. This is the first targeted treatment for melanomas that have NRAS mutations. BRAF and NRAS mutations can activate a protein called MEK that is involved in cell division. The experimental drug, which is called MEK162, is a MEK inhibitor. The side effects of MEK162, which included diarrhea, rashes and swelling, were manageable.

An early stage clinical trial suggests that dabrafenib, a BRAF inhibitor, could treat melanomas that have spread to the brain. The study, reported in The Lancet, included 10 people with brain metastases of melanomas that had BRAF mutations. Tumors shrank in 9 patients and were not evident in 4 patients. This is a surprise because the drug had not been expected to cross the blood-brain barrier effectively. Indeed, melanoma patients with brain metastases have been routinely excluded from previous trials of vemurafenib (Zelboraf) and other BRAF inhibitors.

A New England Journal of Medicine study found that vemurafenib, which was approved by the FDA in 2011, controlled melanomas in about half of people who had been previously treated for this disease. The trial included 132 repeat patients; tumors shrank in 47% of the patients and were not evident in 6% during the course of the trial. Vemurafenib is a BRAF inhibitor and about half of melanoma patients have BRAF mutations. While 26% of patients developed another kind of skin cancer called squamous cell carcinoma, these lesions were successfully removed surgically.

A New England Journal of Medicine study reports a promising new approach to treating melanomas with BRAF mutations, which often respond to BRAF inhibitors for just a short time. Melanoma patients treated with trametinib were stable (ie, did not get worse) for three times longer than those treated with dacarbazine, a conventional chemotherapy drug (4.8 vs. 1.5 months, respectively). Trametinib inhibits MEK, a protein that is activated by BRAF and is involved in cell division. The drug’s most common side effects were rash, diarrhea, and swelling in the legs, which could be controlled by periodically adjusting the dose.

Preliminary results suggest that an imaging technique can give early signs of drug resistance in melanomas. A Journal of Clinical Oncology study found that positron-emission tomography (PET)/computed tomography (CT) scans correlated with standard measures of tumor response in seven melanoma patients treated with vemurafenib. The scans also showed that during the third and fourth weeks of treatment, tumors in three patients began to take up and metabolize more of a sugar. This is a sign of cell activity, suggesting that these tumors were starting to resist the drug.